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Showing 1–5 of 5 results
Advanced filters: Author: "G Dickson" Clear advanced filters

  • Oculopharyngeal muscular dystrophy is caused by trinucleotide repeat expansions in thePABPN1gene. Here the authors use AAV-based gene therapy to knockdown the mutant gene and replace it with a wild-type allele, and show effectiveness in mice and in patient cells.

    • A. Malerba
    • P. Klein
    • G. Dickson
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-14

  • Duchenne muscular dystrophy is a progressive degenerative disease of muscles caused by mutations in the dystrophin gene. Here the authors use AAV vectors to deliver microdystrophin to dogs with muscular dystrophy, and show restoration of dystrophin expression and reduction of symptoms up to 26 months of age.

    • Caroline Le Guiner
    • Laurent Servais
    • George Dickson
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-15

  • It is time to weigh up the pros and cons of using genetic engineering to rescue species from extinction, say Michael A. Thomas and colleagues.

    • Michael A. Thomas
    • Gary W. Roemer
    • Jason Malaney
    Comments & Opinion
    Nature
    Volume: 501, P: 485-486

    • Ian R. Graham
    • Anna Manzano
    • Jim S. Owen
    Correspondence
    Nature Biotechnology
    Volume: 19, P: 507-508